These two reports suggest to me a big future role for preventative gene therapy. The first report provides preliminary evidence that gene therapy can lessen the symptoms of Parkinson's Disease.
MANHASSET, NY -- A novel gene therapy technique is safe and may be effective at staving off worsening symptoms of Parkinson's disease, according to the first scientific review of a dozen patients who have received the treatment over the last three years. The results were published in the latest issue of Lancet.
The patients, half of whom live on Long Island, are in advanced stages of the illness and were no longer responding to medicines when they signed on for the experimental therapy. The study was conducted by Andrew Feigin, MD, director of Neuroscience Experimental Therapeutics at The Feinstein Institute for Medical Research and his colleagues in collaboration with Parkinsonís scientists at New York Presbyterian Hospital-Weill Cornell Medical Center in Manhattan.
One woman and 11 men received a surgical infusion of fluid containing a viral vector and genes for a protein called GAD, glutamic acid decarboxylase. This enzyme is critical in controlling a neurotransmitter called GABA. In Parkinsonís, GABA is reduced in an area of the brain called the subthalamic nucleus. This region is working on overdrive in the disease process and GABA is an inhibitory transmitter and is important in trying to calm this hyper-reactive circuit.
This particular gene therapy does not fix the genes that are contributing to the development of Parkinson's Disease. But the good news is that the therapy appears to successfully deliver genes into brain cells.
The gene therapy would be used to reduce symptoms and not alter the underlying disease process. Finding novel therapies are key as many Parkinsonís patients stop develop complications after prolonged use of traditional medicines.The Feinsteinís David Eidelberg, MD, took brain scans before, during and after the treatment and the scans show that the brain is re-working these abnormal circuits. Dr. Feigin said that patients had about a 27 percent improvement in symptoms, although the study was an open label design. The scientists are now designing a double-blind placebo controlled trial that would enroll far more patients in an attempt to see whether the gene therapy is effective in reducing symptoms.
A far better gene therapy would target the genes that contribute to the development of Parkinson's in the first place. Well, a Mayo Clinic group used the rapidly falling costs of genetic testing to look at lots of genes in Parkinson's sufferers and they found combinations of genetic variations highly predictive for the development of Parkinson's Diseases.
ďBy examining a large cluster of related genes, we found patterns that make people up to 90 times more likely to develop Parkinsonís than the average person,Ē says study co-author Timothy Lesnick, a Mayo Clinic biostatistician. ďThe size of the effects that we observed for genes within a pathway and the statistical significance of the predictive models were unprecedented.ĒThe models were highly effective in predicting age of onset of the disease: by age 60, 91 percent of patients in the highest-risk group already had Parkinsonís, while only 11 percent of patients in the lowest-risk group did. By age 70, every member of the highest-risk group had the disease, whereas two-thirds of patients in the lowest-risk group still were disease-free. Members of the highest-risk group typically developed Parkinsonís more than 20 years earlier than the lowest-risk group.
These two reports are a great example of why personal genome sequencing will become useful. Once brain gene therapy becomes a safe reliable technology anyone with a genetic profile which puts them at high risk of Parkinson's Disease (or Alzheimer's or assorted other brain disorders) will be able to get gene therapy decades before they develop clinical symptoms of a brain disorder.
Preventative gene therapy. That's what I want. Only, I want it for every cell in the brain and for every single thing that ages in the brain. Plus, I want it for the rest of the body. Then we can stop and turn back the clock of biological aging and become youthful again.
The development of gene therapy treatments for most of the maladies of old age (and probably all the neurological maladies) will eventually provide us with most of the tools we'll need for full body rejuvenation. Development of stem cell therapies and techniques for growing replacement organs will provide most of the other tools we need to turn back the aging clock.
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